Mini -dystrophin nucleic acid and peptide sequences
AssigneeThe Regents of the University of Michigan
InventorsJeffrey S Chamberlain, Scott Q Harper
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Abstract
The present invention relates to compositions and methods for expressing mini-dystrophin peptides. In particular, the present invention provides compositions comprising nucleic acid sequences that are shorter than wild-type dystrophin cDNA and that express mini-dystrophin peptides that function in a similar manner as wild-type dystrophin proteins. The present invention also provides compositions comprising mini-dystrophin peptides, and methods for expressing mini-dystrophin peptides in target cells.
Claims34
A composition comprising nucleic acid encoding a mini-dystrophin peptide, wherein said mini-dystrophin peptide comprises a spectrin-like repeat domain comprising n spectrin-like repeats, wherein said mini-dystrophin peptide contains no more than n spectrin-like repeats, and wherein n is an even number that is less than 24 and at least 4.
A composition comprising a mini-dystrophin peptide, wherein said mini-dystrophin peptide comprises a spectrin-like repeat domain comprising n spectrin-like repeats, wherein said mini-dystrophin peptide contains no more than n spectrin-like repeats, and wherein n is an even number that is less than 24 and at least 4.
A composition comprising nucleic acid encoding a mini-dystrophin peptide, wherein said mini-dystrophin peptide comprises i) a spectrin-like repeat domain comprising 4 dystrophin spectrin-like repeats, ii) an actin-binding domain, and iii) a β-dystroglycan binding domain;
- and wherein said mini-dystrophin peptide contains no more than 4 dystrophin spectrin-like repeats.
A method, comprising;
- a) providing;
- i) a vector comprising nucleic acid encoding a mini-dystrophin peptide, wherein said mini-dystrophin peptide comprises a spectrin-like repeat domain comprising n spectrin-like repeats, wherein said mini-dystrophin peptide contains no more than n spectrin-like repeats, and wherein n is an even number that is less than 24 and at least 4, and
- ii) a subject comprising a target cell, and
- b) contacting said vector with said subject under conditions such that said mini-dystrophin peptide is expressed in said target cell.
A composition comprising nucleic acid, wherein said nucleic acid encodes a mini-dystrophin peptide, and wherein said mini-dystrophin peptide comprises a substantially deleted dystrophin C-terminal domain.
Dependencies are inferred from the claim text.